Friday, December 31, 2010

Hepatitis C treatment Eagerly anticipated drug approvals in 2011 include a decision by the US Food and Drug Administration on telaprevir, which could provide relief for the 3% of the world's population infected with the hepatitis C virus. The drug was developed by Vertex Pharmaceuticals in Cambridge, Massachusetts.

In October this year the AASLD annual meeting was held in Boston , MA . The key highlight of this meeting was the release of clinical data for emerging novel direct acting agents (DAAs) for treatment of chronic Hepatitis C virus (HCV) infection.

This year marked an important milestone as Phase III data for two late-stage drug candidates were presented at this meeting.

Other highlights of this meeting were Phase 1 clinical data for new class of drugs and genomic data for IL-28B marker. Based on our attendance at this meeting we have summarized here some of the highlights.

Two promising protease inhibitors for HCVTelaprevir and boceprevir are peptidomimetic molecules which inhibit NS3-4A protease. They are in late-stages of development for treatment of chronic Hepatitis C. Their Phase III data was released at this AASLD meeting.

The use of these agents in combination with peginterefeon and ribavarin has shown up to 75% SVR in TN, and 30% to 85% SVR in TE genotype 1 patients.This triple combination could also reduce treatment duration from current 48 weeks to 24 weeks or shorter, depending upon patient’s early viral response.Based on the history of several drug launches, these two drugs would represent major advancement to the current standard of care.

Personalized medicine in HCVIt seems like that treatment for HCV is setting the stage for one of the best examples of personalized treatment using genomic host data. In the past, patients were tested for genotype of their virus, whether it is 1, 2, 3 or 4. From this year’s presentations it seems like that now patients need to be tested for many other types of genomic data, such as their sub-type (1a versus 1b) and IL-28B alleles. Though in early stages, these data are likely to be used for customizing therapy for individual patients.

Another good news for HCV patients is the possibility of shorter duration of treatment. Clinical studies with novel DAAs are showing that for almost two-third of the patients, their treatment duration can be reduced to 12 or 24 weeks, if they achieve rapid virological response (RVR). Until now, one year treatment with Peg-Interferon and ribavarin has been problematic for several patients, either due to intolerability of interferon, side effects or in-general adherence to one-year regimen. If treatment duration could be reduced to half or one-third time, it would enable many more patients to initiate and complete treatment to achieve SVR.

What factor would drive personalized treatment in HCV?

Testing for sub-types of HCV , IL -28B alleles and customizing based on RVR.What are the challenges in personalized treatment? One challenge is the need for infrastructure to measure and collect all this data, provide individualized guidance and treatment to patients. It is estimated that ~80% of 3 million chronically infected hepatitis C patients are un-diagnosed. As more patients are diagnosed and initiate treatment it would create need for resources to provide personalized care.

According to one potential treatment algorithm presented at this meeting, treatment naïve patients with IL-28B CC allele and fibrosis stages F3-4, could be treated now with Peg IFN+RBV, whereas patients with IL-28B TC/TT allele and fibrosis stages 0-2, could wait for DAAs.

One common theme at this AASLD: “IL-28B”There were more than fifty studies focusing on IL-28B. This marker has gained enormous interest since last year’s meeting.

IL-28B is a gene that codes for Interferon lamba and it has been shown that there is strong correlation between its alleles (TT, TC or CC) and SVR with standard of care.What’s new at this year’s meeting for IL-28B? One is more data from other retrospective studies showing that IL-28B alleles have strong correlation to response. In the future, IL-28B genotype testing can be potentially used to predict outcomes and hence customize regimens for various patients.

Are doctors using it?

Interestingly, answer seems to be yes. In one session three prominent investigators mentioned that they are testing all of their patients for IL-28B genotype. Though its not clear how it will be exactly used in treatment decision making, but doctors admitted that since there is data showing its correlation to response, they have to do testing, for clinical and liability reasons.From this year’s presentation its looks like IL-28B is going to be important in the future for clinical trial design, patient stratification and clinical decision making

Outstanding challenges in treating Hepatitis C patientsWhile there are several promising therapies that are on the horizon, there are still several challenges facing treatment for HCV patients.

First challenge is screening and diagnosing these patients before it is too late to treat them. At this meeting, CDC presented their proposal to screen patients based on birth-year cohort (~65% of HCV patients were born during 1945-1965). How soon and effectively we can diagnose these patients during next 2-5 years is an unknown at this stage.

Patient education could be a challenge, as more genomic and response data would be used to customize therapy for patients. For the near term it seems like that with telaprevir and boceprevir, patients will have high pill burden. I think it would be valuable to have patient counselors who can educate and guide HCV patients and keep them adherent on triple combinations. In HIV, patient survivors have been very successful in acting as counselors for newly diagnosed patients. I would recommend state and federal agencies and drug manufacturers to train HCV-survivors as counselors for these baby-boomers who would receive treatment during next 2-5 years.

Cost of treatment: This could be an issue for low-middle income patients, especially if they have to pay 10-20% of cost for triple combination regimen.

Payers: Several investigators raised the issue that in the future payers might play a role in determining whether patients should continue one versus another regimen. This is especially possible for patients who do not achieve RVR and would have only 4-5% chance of achieving SVR with complete regimen.

Lead in or no lead in: Two different strategies were used for Phase III clinical trial design for telaprevir and boceprevir. One used RVR with triple combination, while other used Lead-in with Peg-infereon and ribavarin, followed by triple combination. Which one is better? How do we define better? Is it based on efficacy, safety, cost, patient genotype or patient convenience? I think in this case all of them would be important.

Despite the significantly high efficacy of telaprevir and boceprevir there is still large unmet need for newer therapies in HCV. What are those areas?

Well, immediate ones are:

Treatment options for genotype 1 null-responsesTreatment options for patients who would fail on new triple combinationsNew therapies are needed for genotype 4 patientsStrategies to reduce pill burden with triple combinationInterferon free regimens

#5 Hepatitis C Protease Inhibiting DrugsHepatitis C, a common liver disease that affects an estimated 3.9 million people in the United States, is transmitted through exposure to infected blood (blood was not screened for hepatitis C until 1992) and sexual contact with an infected person. The majority of people with the ailment have no bothersome symptoms, which is why health experts say millions are unaware that they even have it.

Hepatitis C is typically diagnosed when abnormal liver enzymes are identified through a routine blood test or if the infection becomes severe. Seventy percent of patients with hepatitis C develop chronic disease and 30% may develop cirrhosis of the liver within 20 years of exposure to the virus, which causes severe scarring of the liver. An additional 20% of these patients eventually develop liver cancer.

Hepatitis C virus infection is the leading cause of chronic liver disease, the reason for more than 30% of liver transplantations, and a major contributor to as many as 12,000 deaths annually. Total medical costs for people with hepatitis C are approximately $30 billion.

To date, treatments for hepatitis C virus infection have been somewhat disappointing. The best cure rates following a 24-to 48-week course of therapy with the combination of the oral antiviral medicine ribavirin (taken three times a day) and an injectable interferon (once a week) is about 40%. The flulike side effects of these drugs are often debilitating, however, leading many patients to stop treatment. When a second round of treatment is used with this chemotherapy combination, success rates plummet to 10%. There are an estimated 300,000 Americans with hepatitis C who have failed this treatment protocol. When medical care is ineffective and the disease progresses, a liver transplant may be necessary.Unfortunately, there are no proven medicines for patients who don't respond to traditional hepatitis C therapyâ€”until now, that is.

Two advanced drugs called hepatitis C protease inhibitors now awaiting approval from the Food and Drug Administration (FDA) have the ability to fundamentally change the treatment for hepatitis C for patients who have not responded to previous therapies. Both new drugs have been very successful in curing test subjects in clinical trials, which represents a big step forward in successfully battling this debilitating disease.

The experimental drugs belong to a class of medications called protease inhibitors, which work by blocking a key enzyme that viruses need in order to copy themselves and proliferate. New data from Phase 2 trials report that when the experimental drug telaprevir, developed specifically to target hepatitis C virus, was added to standard treatments for hepatitis C, 72% of the patients achieved sustained viral response (or viral cure) after taking the medication for 24 weeks. This study confirmed that the 24-week course was just as effective as it was for patients taking the drug cocktail for 48 weeks.

Study results with a drug called boceprevir were similar with difficult-to-treat patients with hepatitis C, although cure rates were slightly lower with the three-drug combination. When combined with interferon and ribavirin, boceprevir cured the infections of about 66% of the patients who took the drugs for 48 weeks compared to 38% of those in the control group who received the standard therapy for 48 weeks.The good news is that cure rates with these new protease inhibitors are higher than those with standard hepatitis C therapy. And so, after decades of research and expected FDA approvals, a new era of antiviral medications developed specifically to target hepatitis C virus is about to begin.

Our Year in Review series highlights the major medical news stories of 2010. The risks associated with dietary supplements was one of the compelling topics that made headlines during the year and here, again, is the original article, first published on Nov. 17, 2010. In a companion article, you'll find out what's happened since.

Updated: Jan 6thThe U.S. Food and Drug Administration accepted Merck's approval filing for its hepatitis C drug boceprevir, the company said Thursday. The news from Merck is a bit of a surprise since the company hadn't previously announced or confirmed that boceprevir had been filed with U.S. regulators.It's also a tad embarrassing for Vertex, which is still waiting to hear back from FDA about the acceptance of the approval filing for telaprevir, its competing hepatitis C drug. That news should come by Jan. 24, based on Vertex's previous announcement that it filed the drug's application on Nov. 23.Still, it seems as if Merck beat Vertex to the FDA by at least two weeks.Continue Reading....Press Release.....

Will the National Ignition Facility ignite 2011?LBNLThe Eemian revealed

The North Greenland Eemian Ice Drilling (NEEM) project reached bedrock in July 2010, at a depth of more than 2,500 metres. The fruits of that effort should soon be seen, now that researchers are analysing gas and particles trapped inside the ice core to reveal details of the climate of the Eemian interglacial period (130,000–115,000 years ago), when the average global temperature was about 5°C warmer than today.

GWAS prove their worth Genome-wide association studies (GWAS) have uncovered plenty of links between diseases and particular regions of the genome, but frustratingly haven't revealed much about the biochemistry behind these associations. In 2011, expect to see real mechanistic insights explaining how genes, and non-coding regions, affect the medical conditions they have been linked with. Metabolism, obesity and diabetes are among the hottest targets.Stem cells: ready for study Researchers have learned how to reprogram people's cells into induced pluripotent stem (iPS) cells, and on from that into other cell types: skin cells can be turned into nerve cells, for example. Patient-derived iPS cells will increasingly be used as models for studying medical conditions — particularly those, such as psychiatric disorders, for which there are no good animal models, and little understanding of what is happening inside cells. They will also be used to screen potential drugs, and to probe why existing drugs help some patients but not others.

Genome-sequencing explosion This year should surely see the price of human-genome sequencing dropping to US$1,000 per genome. As next-generation sequencing machines reach the market, the number of fully sequenced human genomes will skyrocket.That damned elusive Higgs Although it is unlikely that the Higgs boson will be spotted this year by the Large Hadron Collider near Geneva, Switzerland, there's a good chance the collider will turn up something, such as evidence for supersymmetry — the theory that every known fundamental particle has an undiscovered, superheavy partner. Meanwhile, Fermilab's Tevatron in Batavia, Illinois, is pushing for an extension beyond its September 2011 shutdown, and still hopes to hit the Higgs jackpot.

Dark matter's moment of truth A number of underground experiments, such as XENON100 at Italy's Gran Sasso National Laboratory near L'Aquila, and the Cryogenic Dark Matter Search (CDMSII) in northern Minnesota's Soudan Mine, are hunting for dark matter particles and expect to release results in 2011.

Hepatitis C treatment Eagerly anticipated drug approvals in 2011 include a decision by the US Food and Drug Administration on telaprevir, which could provide relief for the 3% of the world's population infected with the hepatitis C virus. The drug was developed by Vertex Pharmaceuticals in Cambridge, Massachusetts.

Another Earth Planet-hunters anticipate that NASA's Kepler telescope will reveal an Earth-like planet orbiting a Sun-like star. It has already spotted hundreds of planets outside the Solar System, although full data have not yet been released.

Synthetic biology: think multicellular No longer will scientists have to cram compli­cated synthetic biology into a single cell. Last year, researchers engineered an entire colony of bacteria to periodically fluoresce in unison, and we can expect many more papers exploring the behaviour of collections of cells. The goal is to exploit this teamwork to give bacteria useful functions such as producing medicinal drugs.

Last of the shuttles The final flight of NASA's space-shuttle fleet is scheduled for April, when it will deliver the Alpha Magnetic Spectrometer (AMS) to the International Space Station to search for antimatter and dark matter. However, the US Congress may authorize another shuttle outing in November. If the second test launch of Dragon, the craft developed by commercial spaceflight firm SpaceX in Hawthorne, California, proves successful, the launch of a private spacecraft with crew or cargo is not out of the question.

Solar-system explorers In March, NASA's Messenger mission is due to become the first craft ever to orbit Mercury, and the agency's Dawn probe will orbit one of the biggest members of the asteroid belt, Vesta, in August. Other planned space launches include Juno, which will orbit Jupiter's poles; the GRAIL mission, twin spacecraft due to measure the Moon's gravitational field; and the Mars Science Laboratory, a car-sized rover that will explore the red planet.Superlaser flirts with fusion California's National Ignition Facility (pictured), the world's most power­ful laser, is inching its way to triggering ignition, when fusion reactions in a target of hydrogen isotopes should produce more energy than the laser delivers. Experts give even odds that the laser, at the Lawrence Livermore National Laboratory, will succeed this year.Probing home GOCE, a NASA satellite designed to measure Earth's gravity field in unprecedented detail, will publish results next year that will be used to help monitor sea level rise. Meanwhile, the Aquarius satellite will launch to measure ocean salinity, and Glory will monitor solar irradiance and aerosols.

EHSI Secures Rights to Use NASA Developed Stem Cell Technology in ChinaNASA Developed Bioreactor Could be Huge Boost for Company’s Celulas Genetica Subsidiary

The Rutherford Procedure is a groundbreaking organ regeneration treatment that utilizes proton-beam technology to destroy diseased organ tissue for regeneration using adult stem cells. During the procedure, proton therapy will be used to destroy scar-tissue cells in the liver using high-energy proton beams, a non-invasive treatment proven to minimize damage to healthy tissues and to eliminate the side effects (including nausea) of traditional radiation therapy. As the scar tissue is systematically destroyed by the proton therapy, a catheter will deliver the patient’s own cultured stem cells directly to the patient’s liver through the bloodstream. As more and more diseased tissue is destroyed, these cultured stem cells could help regenerate the patient’s damaged, cirrhotic liver into a healthy, functioning organ once more.

December 30, 2010 09:15 AM Eastern TimeHOUSTON--(EON: Enhanced Online News)--Emerging Healthcare Solutions, Inc. (PinkSheets:EHSI) revealed today that their new License Agreement with Regenetech specifically allows the Company to use NASA’s Intrifuge Rotary Cell Culture System in The People’s Republic of China. EHSI has been granted a sub-license for the NASA Intrifuge Rotary Cell Culture System from Regenetech in the License Agreement. What differentiates the NASA technology is the addition of simulated weightlessness in the Rotary Cell Culture System or “bioreactor”. Cell cultures, including stem cells, grown inside the bioreactor look and function much closer to human cells grown within the body than cell cultures grown in Petri dishes. EHSI has also purchased an Intrifuge Rotary Cell Culture System.

This new license is essential to new stem cell research planned by Celulas Genetica, EHSI’s biotech division, including its current endeavor to develop a revolutionary new cure for liver disease known as the Rutherford Procedure. Celulas Genetica is currently planning to test this new procedure in China.

The Rutherford Procedure is a groundbreaking organ regeneration treatment that utilizes proton-beam technology to destroy diseased organ tissue for regeneration using adult stem cells. During the procedure, proton therapy will be used to destroy scar-tissue cells in the liver using high-energy proton beams, a non-invasive treatment proven to minimize damage to healthy tissues and to eliminate the side effects (including nausea) of traditional radiation therapy. As the scar tissue is systematically destroyed by the proton therapy, a catheter will deliver the patient’s own cultured stem cells directly to the patient’s liver through the bloodstream. As more and more diseased tissue is destroyed, these cultured stem cells could help regenerate the patient’s damaged, cirrhotic liver into a healthy, functioning organ once more.

Celulas Genetica purchased a license to develop and market the Rutherford Procedure earlier this month, a day before EHSI announced the company’s acquisition. Celulas Genetica is only the latest outpost in Emerging Healthcare Solutions’ global footprint. In addition to its Houston headquarters, the company also maintains business offices in Frankfurt, Germany and Warsaw, Poland.

Emerging Healthcare Solutions, Inc. invests in and participates in the profits of emerging breakthrough medical technologies. The Company believes the secret of leveraging future value for its shareholders is the proper timing of its investment in promising new medical technologies. EHSI aims to capture future profits of promising new medical technologies by investing in these technologies at the inflection point of product development. We believe this model will deliver long-term positive results for our investors.

For more information about EHSI, please visit www.EmergingHealthcareSolutionsInc.com.

Safe Harbor Statement under the Private Securities Litigation Reform Act of 1995: This news release contains forward-looking information within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, including statements that include the words "believes," "expects," "anticipate" or similar expressions. Such forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause the actual results, performance or achievements of the company to differ materially from those expressed or implied by such forward-looking statements. In addition, description of anyone's past success, either financial or strategic, is no guarantee of future success. This news release speaks as of the date first set forth above and the company assumes no responsibility to update the information included herein for events occurring after the date hereof.

Doctors have started the largest trial of its kind in the world to see if stem cells can repair damaged livers.

Eighty patients will be treated in Birmingham and Edinburgh to analyse whether the use of targeted stem cells can reduce the amount of scarring or cirrhosis of the liver.

It is also hoped that they can get the liver's own cells to divide creating a benefit for the patient.

In the trial, patients will be given injections of the drug GCSF for five days.

This stimulates adult stem cells, which are normally found in bone marrow, to multiply at a much faster rate so that they spill out into the blood stream.

A machine is then used to collect the cells from the blood.

Patients who are displaying symptoms of liver cirrhosis are involved in the trial Once harvested, the cells are purified, so that a high concentration of the right type of stem cells can be injected back into the patient's blood stream.

The Repeated Autologous Infusions of Stem Cells in Cirrhosis, or 'Realistic' trial, will compare the current standard treatment to both the effect of giving GCSF injections on their own and giving the injections, collecting the stem cells and putting them back into the bloodstream.

Dr Philip Newsome, from the Centre for Liver Research at Birmingham University, is the clinical leader for the trial.

He said that liver disease was increasing, partly due to the obesity epidemic.

New treatments are needed because a liver transplant is currently the only treatment that will improve a patient's condition.

"We know that when the liver is injured, it changes the molecules on the surface of the liver to attract these particular stem cells," he said.

"So by giving patients the drug, GCSF, not only does it put the stem cells into the blood circulation, but it also makes them more likely to go to the liver where we think that they can help break down scarring and also get the liver's own cells to divide.

"When there's a lot of scarring or cirrhosis of the liver, the liver is unable to overcome the damage. It needs a boost and an injection of stem cells allows the liver to get that boost."

Flu-like symptoms

The trial is recruiting patients who are just beginning to exhibit symptoms of liver cirrhosis.

Patients such as 41-year-old Iain Broomhill, from Ellesmere Port, Cheshire, whose liver disease is unexplained, are being recruited.

"If it works, it will have been well worth it, but it's not just about the here and now, it's about the future and if they can get results that improve things for patients in the future, then that's a good thing."

What are stem cells? Stem cells are unspecialized cells that have the ability to develop into cells with specific functions—for example, muscle cells or red blood cells.The two main types Stem cells are found both in embryos and in certain tissues. Embryonic stem cells develop into all the different types of cells the body needs. Stem cells found in tissues serve to repair damage and replace old cells.
Most human embryonic stem cells come from embryos that are four or five days old and still microscopic.Similarities and differences

• All stem cells can divide many times without turning into specialized cells. In a laboratory, a small batch of stem cells can multiply over many months and yield millions of cells.
• Embryonic stem cells can develop into any of the more than 200 types of cells found in the human body.
• Embryonic stem cells can be produced fairly easily in the laboratory, by cell division.
• Adult stem cells (also called somatic stem cells) are found in specific tissues of the body. Usually, they develop only into the type of cells needed in those tissues. Adult stem cells have been found in the brain, bone marrow, blood, skeletal muscle, skin, teeth, heart, and liver.
• Adult stem cells are difficult to isolate from the surrounding specialized cells, and scientists haven’t yet learned how to grow them in the laboratory. This makes them less promising for therapies that would involve replacing damaged tissues with tissues grown from stem cells. However, certain adult stem cells are already being used in medical therapy: for example, in bone marrow transplants for leukemia patients. (Bone marrow contains stem cells that produce blood cells.)Why are researchers so excited about stem cells?

Stem cells offer the hope that doctors will one day be able to replace damaged cells with new, healthy ones derived from the same person—thereby avoiding the problem of tissue-rejection. (Patients who receive transplants must take anti-rejection drugs, which have unwanted side-effects. And the need for organs and tissues to transplant far exceeds the supply.) Millions of Americans suffer from diseases that may some day be treated with stem cell therapy.The first medical therapy using embryonic stem cells was approved by the U.S. Food and Drug Administration in March, 2010. The treatment is for a type of blindness known as Stargardt’s Macular Dystrophy.Scientists also hope to use stem cells to study the effectiveness of new drugs: experimental medications could be tested on tissue grown from stem cells, rather than on human subjects. And stem cell research helps advance our understanding of how adult organisms develop from a single fertilized cell. Finally, scientists hope to learn more about cancer and birth defects by studying stem cells.What diseases and injuries may some day be treated with stem cells?

Among others, Type 1 diabetes, heart disease, spinal cord injuries, vision loss, hearing loss, Alzheimer’s disease, Parkinson’s disease, multiple sclerosis, A.L.S. (Lou Gehrig’s disease), stroke, burns, and arthritis.
The ethical debate In order to extract human embryonic stem cells, the embryo—which has the potential to develop into a human being—must be destroyed. This is the crux of the ethical debate.Most of these embryos come from fertility clinics. Many couples having difficulty conceiving children opt for in vitro fertilization, in which sperm and egg are fused in a laboratory; a resulting embryo is then implanted in the mother’s uterus. Several eggs are usually fertilized, but only one is implanted in the mother. With the consent of the parents, the extra embryos are donated for research.Proponents of research using embryonic stem cells say that the potential medical advances—including treatments for diseases that doctors can’t cure at present—far outweigh the ethical concerns. They also point out that the excess embryos would be destroyed anyway if they weren’t used for research.
Opponents say that this research disrespects human life. (Most of these opponents also oppose abortion.) The Catholic Church is the most prominent opponent of stem cell research using human embryos.There is less controversy over research involving adult stem cells, because no embryo is destroyed in extracting them. The Catholic Church supports this type of research.
For a more detailed exploration of the ethical issues of embryonic stem cell research, go to ReligiousTolerance.org. For more on the political controversy, go to Newsbatch.com

Milestones in stem cell research1960s While studying rats, scientists discover cells in the brain that divide and eventually become nerve cells.
1981 Researchers learn how to derive stem cells from mouse embryos.
1998 Researchers at the University of Wisconsin-Madison extract the first stem cells from human embryos that can be kept alive in the laboratory.
2006 Researchers learn how to induce some specialized adult cells to return to an unspecialized, stem-cell state.
2010 U.S. doctors begin the first official trial using human embryonic stem cells in patients. The trial, at an Atlanta hospital, involves patients with spinal injuries.
(See this BBC story for more information.)What research is currently allowed by law? A 1996 law known as the Dickey-Wicker amendment bans the use of tax dollars to create human embryos, or to conduct research in which human embryos are destroyed, discarded, or injured. That law prevented federal funding of embryonic stem cell research—until August, 2001, when President George W. Bush found a compromise by ordering that tax dollars could be used for research on the 60 stem cell lines that already existed. (A stem cell line is a population of healthy stem cells, derived from a single parent group of stem cells, that have divided for at least six months without differentiating into specialized cells.)In March, 2009, President Obama removed the limits set by President Bush. His executive order will allow research on all embryonic stem cells—but federally-funded researchers still won’t be allowed to create their own stem cell lines.
Some countries, including Austria, Denmark, France, Germany and Ireland, have banned the production of embryonic stem cell lines. Countries that permit it include Finland, Greece, the Netherlands, Sweden, and the United Kingdom.

Recent news
• 8/26/10: British scientists create liver cells from stem cells taken from human skin, by “reprogramming” the skin cells. This seems to support hopes that healthy cells can someday be used to repair damaged organs—and that adult stem cells can be used in place of controversial embryonic stem cells. See Yahoo News for more.
• 8/24/10: In a surprise to researchers, the New York Times reports, “a federal district judge blocked the president’s 2009 executive order that expanded embryonic stem cell research, saying it violated a ban on federal money being used to destroy embryos.”

Terms to knowblastocyst: an embryo that’s 3 to 5 days old. A hollow, microscopic ball, the blastocyst consists of 50-150 cells. Its inner cells eventually develop into the entire adult body. Blastocysts are the source of human embryonic stem cells.
cell-based therapies: medical treatments using stem cells to generate replacements for injured or diseased tissue.
cell culture: growing cells in a laboratory.
differentiation: the process by which an unspecialized cell develops into a specialized one.
directed differentiation: inducing embryonic stem cells to turn into specific cell types. Scientists have learned how to create a few different cell types this way.
embryo: in humans, a fertilized egg up to the age of eight weeks. (After eight weeks, it’s called a fetus.)
pluripotent: able to develop into any type of cell in the body.
induced pluripotent stem cells: cells that have been reprogrammed to behave like embryonic stem cells, by implanting embryonic genes into them.
regenerative medicine: using cell-based therapies to treat disease.transdifferentiation: the differentiation of adult stem cells found in one type of tissue into cells of a different type. This has been observed in some species, but scientists disagree on whether it happens in humans. (Note: Scientists have recently “reprogrammed” certain types of adult cells into other types of cells through genetic modification.)
Why are they called stem cells? Think of the stem of a plant: from one stalk, many smaller limbs branch off. Similarly, from one stem cell, many different types of specialized cells develop.

Opinions
For an overview of ethical concerns on the use of embryonic stem cells, see ReligiousTolerance.org.
Stem Cell Opinion Roundup, Biopolitical Times (website), 3/11/09: opinion and commentary on the change in federal human embryonic stem cell research policy as ordered by President ObamaTwo letters to the editor in the New York Times, published 8/26/10, sum up the opposing positions on stem cell research:
Arthur L. Yeager, Edison, N.J.: “By what distorted sort of moral values are thousands of frozen embryos, no longer needed by couples for in vitro fertilization and about to be thrown into the medical waste trash bin, prohibited from being used for vital research to cure diseases like diabetes, cancer, heart disease and Parkinson’s, to find remedies for debilitating injuries and to promote vital organ regeneration?”Paul Kokoski, Hamilton, Ontario: “The American people should not be forced to pay for experiments that destroy human life. Human life begins at conception with the formation of a genetically complete, self-directing human entity, the embryo. Human beings are not raw materials that can be exploited or commodities that can be bought and sold. We must help those who are suffering, but we may not use a good end to justify an evil means. The respect for every human life is an essential condition of our society. Any method of genetic manipulation that involves the alteration or destruction of human embryos is nothing more than Frankenstein science.”

'Hospitality' taken to new levels / Vet helps set up medical centers for Mongolians with hepatitis

Takashi Nakamura / Yomiuri Shimbun Staff Writer

Nobuyuki Nozawa has been fascinated by Mongolia since he was a child--particularly its people's nomadic lifestyles--but not even he could have foreseen he would end up helping combat viral hepatitis in Ulan Bator.

The 55-year-old veterinarian has helped establish two facilities there to detect and treat viral hepatitis, with the most recent, the Interferon Alpha Hospital, opening earlier this month.

A resident of Arakawa Ward in Tokyo, Nozawa first visited Mongolia in 1988. "[Mongolians] live such interesting lives. Their homes are borderless, beyond horizons. They're deeply attuned to nature and their horses. They ride them as if they were bicycles," he said.

He began making regular visits and registered with a mailing list for Japanese interested in and knowledgeable about the country.

It was through this mailing list that in 1999 he first met Mongolian doctor Jazag Amarsanaa.

Amarsanaa was studying liver and digestive diseases at the University of Tokyo and looking for part-time work. Amarsanaa remained in Japan until 2006, when he headed to the United States for further study. He returned to Mongolia in 2008.

From Amarsanaa, Nozawa learned of the severity of viral hepatitis in Mongolia: About 10 percent of Mongolians are infected with hepatitis B and 10 to 35 percent with hepatitis C. Amarsanaa also told Nozawa he wanted to set up a facility to detect viral hepatitis.

Nozawa and many others contributed money to establish the Happy Veritas medical center which opened two years ago.

Happy Veritas has since tested about 13,000 people for hepatitis; of those, about 70 percent tested positive for either the B or C strains.

It soon became apparent a hospital was also needed to treat hepatitis patients, and Nozawa invested 100,000 dollars (about 8.2 million yen) to help open Interferon Alpha Hospital in the same building as Happy Veritas.

The hospital, which opened on Dec. 16, has 10 staff and 12 beds. Amarsanaa is its president.

Nozawa hopes the establishment of the two medical facilities will help Mongolian people detect hepatitis during its early stages and receive the appropriate treatment.

"By helping hepatitis sufferers in Mongolia, I hope to make as many people as happy as possible," he said.

The Centre for Food Safety (CFS) warned of rise in cases of Hepatitis E in Hong Kong and appealed to citizens to cook food more thoroughly, particularly pig livers.

"The number of reported cases of Hepatitis E has been climbing since 1998. It's more than 100 in 2010, the highest ever in the city," said Ho Yuk-yin, doctor and consultant on Risk Assessment and Communication at the CFS. The number was less than 10 in 1999 and less than 40 in 2006, according to the Centre for Health Protection (CHP).

But the CFS is yet to know why. "It's not just in Hong Kong, but many other areas also, where more locally infected cases have been reported," Ho said, "a lot of scientists are still exploring the reasons."

Hepatitis E viruses are mainly transmitted though contaminated water or food. Symptoms include fever, malaise, anorexia, nausea, dark urine and jaundice. The disease is mild and spontaneously resolves in two weeks, leaving no sequela.

But it could be dangerous for pregnant women and patients with pre-existing chronic liver diseases. The case-fatality rate can reach 70 percent among the latter.

The human cases of Hepatitis E are rare in developed countries. Most of the afflicted are said to have visited endemic-prone developing countries. Yet an increase in sporadic cases has been reported even from those who haven't traveled abroad in recent years, Ho added.

An analysis of 51 human cases in Hong Kong by the CHP in 2008 found 65 of them had never gone to any endemic-prone areas.

The CFS believes that the semi-cooked pork livers could be a major source of Hepatitis E in Hong Kong.

To study a possible relationship between pork livers and Hepatitis E virus, the center collected 100 fresh pig livers from slaughterhouse in 2009, and found none of the 6-month pigs was infected with Hepatitis E virus, while 31 percent of 4-month pigs were infected.

The CFS compared the virus detected from the 48 human cases reported during the first seven months in 2009 with those from pigs and found seven partial matches. "The finding suggests that undercooked pork livers could be a source of human Hepatitis E cases," Ho said.

"It's important for all to cook food more thoroughly to lower the risk," Ho said, noting some people like to eat half-cooked pork livers. "Particularly these people must be more alert. Viruses are more resistant to heat than bacteria," Ho explained.

Ho advised all to boil sliced pig liver at 100 centigrade or stir-fry for at least three to five minutes. For shellfish, either boiling for additional three to five minutes after their shells open, or heating to an internal temperature to 90 centigrade for 90 seconds, is necessary.

Discussion Acute HCV infection is a condition that has hitherto been unrecognized by medicine, as HCV-infected individuals normally present after a lag period of many years following initial infection. Given the rarity of its presentation, this is the first study to assess the effects of acute HCV infection on the central nervous system. We have observed significant reductions in RBG mI/Cr ratio in HIV-1 infected subjects with acute HCV infection, compared with that in matched groups of HIV-1 infected subjects without HCV infection and in healthy controls. Furthermore, significant abnormalities in neurocognitive function were observed.

The fall in mI/Cr ratio that we have observed was predominantly secondary to a reduction in mI in subjects with acute hepatitis C, although there was a trend towards a raised Cr in this group also [mI (±SD) of 121 (±13) vs 129 (±11) and 130 (±10) and Cr (±SD) of 44 (±8) vs 38 (±4), 39 (±5) in groups 1 vs 2 and 3, P = 0.049 and 0.143 respectively]. Other groups have described altered cerebral metabolite patterns in chronic HCV, rather than acute HCV, with differing results including elevation of basal ganglia and central white matter Cho,[2,16,29] reductions in grey and white matter NAA[5,29] and, of note, increases in FWM mI/Cr ratio.[20]

We have utilized the jMRUI software package which uses the AMARES algorithm to analyse our data.[27] This algorithm has several advantages such as the programming of prior knowledge to analyse data which reduces operator-dependent variability.[30] Forton et al.,[20] utilizing a similar analytical method, also observed changes in mI/Cr ratio in patients with chronic HCV, but reported an increase in this ratio in the FWM, rather than the reductions in mI/Cr that we found in patients with acute HCV. mI is an osmosensitive glial marker and plays a crucial role in cell volume regulation.[31] Organic osmolytes, such as mI, accumulate inside the cell in response to cell shrinkage, and are rapidly released in response to cell swelling via osmoregulated membrane channels.[32,33] Our findings are novel in patients with acute viral infections, as a low mI/Cr ratio has previously been observed only in patients with end-stage liver disease with established cirrhosis and hepatic encephalopathy, where there are varying degrees of cerebral oedema in response to the presence of unfiltered circulating toxins, such as ammonia, with subsequent regulatory lowering of mI levels in an effort to maintain astrocyte osmotic equilibrium, as the brain swells.[34] Clearly, this is not the mechanism in our cohort with documented acute HCV infection, who may be up to 30 years away from developing HCV-related end-stage liver disease, if their viral infection persisted. We hypothesize that the reduction in mI/Cr ratio we have observed may be an early response to the direct effects of HCV on the CNS and could suggest an initial cellular swelling as part of the acute neuroinflammatory response to the presence of the HCV.

Previous reports have described significant changes in cerebral metabolite patterns in HIV-1 infected subjects with AIDS Dementia Complex, such as an increase in mI in FWM.[35,36] We did not observe any differences between groups 2 and 3 in our study, which is likely to reflect the exclusion of any baseline neurological disease from inclusion in this study. Furthermore, the mean CD4+ lymphocyte count in the HIV-1 mono-infected group was high at 392 cells/μL (SD ± 124), with 60% of subjects on ART; both factors associated with a reduced incidence of HIV-1 related brain injury.

In HIV-1 non-infected subjects with chronic HCV infection, studies have suggested that up to one-third of subjects have CNS dysfunction.[20] We have observed a mI/Cr ratio below the lowest value in 50% of subjects in our control population. Although small numbers are represented, the relatively high proportion of subjects with evidence of HCV-associated CNS disease may be related to the acute nature of the infection, with a more aggressive inflammatory response, or viral replication, or may reflect a greater propensity for CNS dysfunction in HIV-1 infected subjects. It has been postulated that the effects of HCV on the CNS may be because of cerebral immune activation[37] or direct viral replication.[15] In the context of HIV disease, both of these processes may occur at increased rates.

Chronic fatigue has been well described in chronic HCV infection.[29] In this study, we describe a significant reduction in the monitoring domain with a large effect size of this association (r-value >0.28).[28] This may be associated with fatigue and an inability to concentrate on a task in the setting of acute HCV. No significant associations were observed directly between this cognitive test and cerebral metabolites, which may be related to small study numbers.

Abnormal cerebral metabolism has been documented in recreational drug-dependent subjects[38] and may confound results in studies assessing CNS function in HCV. We therefore excluded subjects currently using recreational drugs, thereby eliminating this bias and we excluded subjects with other sexually transmitted infections which are known to cause CNS disease, such as early syphilis.

This study is the first report to describe significant effects of acute HCV infection on the CNS in HIV-1 infected subjects. Clinicians should be vigilant for the early onset of the CNS effects associated with HCV infection, particularly in those co-infected with HIV. Further studies are required to put these results into context, including cerebral positron emission tomography with the specific ligand [11C](R)-PK11195 as a marker of microglial activation,[39] and there is a need for treatment programmes which monitor viral eradication in acute HCV infection to incorporate assessment of these imaging and psychometric parameters to look for resolution over time of the CNS observations that we have reported.Continue reading..........

WASHINGTON (AFP) –Two US companies this year broke new ground by winning regulatory approval to start the first experiments using embryonic stem cells on humans suffering from spinal cord injury and blindness.

The potent but hotly debated cells can transform into nearly any cell in the human body, opening a path toward eliminating such ills as Parkinson's disease, paralysis, diabetes, heart disease, and maybe even the ravages of aging.

And more human experiments are on the way as scientists refine new methods to get around the controversy that surrounds embryonic stem cell research, which involves the destruction of early human life.

"After a decade of intense controversy, the field is finally ready to start proving itself and to actually start helping patients suffering from a range of horrific diseases," said Bob Lanza, chief scientist at Advanced Cell Technology.

His company was cleared in November by the US Food and Drug Administration to begin testing a therapy derived from embryonic stem cells to treat a rare form of blindness that strikes in childhood, known as Stargardt's disease.

Clinical trials are expected to start in the coming months, and results could be known within six weeks.

In October, Geron Corporation announced it had begun the first-ever test of human embryonic stem cells in a patient suffering from spinal cord injury. In all about a dozen patients are expected to participate in the year-long study.

The primary aim of both ACT's and Geron's studies is to gauge safety, not necessarily to restore mobility or vision.

The major concern with stem cell therapies is that the transforming cells could form tumors. But if the methods appear safe, both companies aim to expand their trials to wider populations in the hopes of eventually curing paralysis and blindness.

Twelve years ago, American scientist James Thomson's team isolated human embryonic stem cells for the first time, and the field has been cloaked in controversy ever since.

Former president George W. Bush outlawed federal funding for the research because it involves the disposal of human embryos, a ban that President Barack Obama reversed shortly after taking office in 2009.

But in August of this year, Judge Royce Lamberth blocked US government funding for embryonic stem cell research after ruling in favor of a coalition of groups, including several Christian organizations.

While the funding has since been permitted to go ahead pending appeal, the legal wrangling has left some scientists wary of the future.

"The on-again-off-again situation will only stall the progress of everyone?s work," said Tim Kamp, head of the University of Wisconsin's Stem Cell and Regenerative Medicine Center, back in September.

To get around the problems associated with embryonic stem cell research, scientists in 2010 forged new paths toward creating induced pluripotent cells, which can transform into skin, blood or heart cells. Embryonic stem cells are pluripotent cells.

The field of induced pluripotent cells (iPS) faces its own challenges, as studies have shown they are less efficient and more unpredictable than embryonic cells.

But Canadian researchers described this year in the journal Nature their method of turning adult human skin cells into blood without manipulating them back into pluripotent cells, making the process more time efficient and potentially safer.

And a Harvard University scientist, Derrick Rossi, discovered a way to avoid risky genetic modification and instead use RNA molecules to reprogram adult human cells into pluripotent cells without altering the DNA.

Describing his peer-reviewed research published in September, Rossi said it was a "safe, efficient strategy... that has wide ranging applicability for basic research, disease modeling and regenerative medicine."

Lanza said the advances, while they still face rigorous testing, offer promise toward treating a host of diseases, and could one day eliminate the need for amputation of limbs, blood transfusions and transplants from strangers.

"Some time in the future, perhaps in the lifetime of most of your readers, you'll get in an accident and lose a kidney and they will take a skin cell and just grow you up a new organ," said Lanza. "That field is just roaring ahead."

If a lot of chemistry work has to be done later and what we put in development is not the original compound, then we take the IP in case the same molecule can be used in a commercial environment. For example, some compounds for dengue might also work for Hepatitis C, which is a blockbuster. We could give that IP to Novartis’ pharmaceutical department, which is a commercial organization...

Idenix Pharmaceuticals to Present at Two Upcoming Investor ConferencesWritten by TradersHuddle Staff Monday, 07 February 2011 16:08CAMBRIDGE, Mass.-( Business Wire )-Idenix Pharmaceuticals, Inc. (NASDAQ: IDIX), announced today that Idenix management will present a corporate overview at the 13th Annual BIO CEO & Investor Conference on February 14, 2011 at 10:30 a.m. ET at the Waldorf Astoria, New York City and at the Leerink Swann Hot Topics Roundtable Conference on February 16, 2011 at 2:20 p.m. ET at the Roosevelt Hotel, New York City. The live and archived webcasts of the company presentations can be accessed under “Calendar of Events” in the Idenix Investor Center at www.idenix.com. Please log in approximately 5-10 minutes before each event to ensure a timely connection. The archived replay will be available on the Idenix website for two weeks following the conferences.

The fourth quarter net loss was $531 million, or 17 cents a share, the Whitehouse Station, New Jersey-based company said. The company withdrew its long-term profit forecast of high single-digit growth through 2013.

In the past quarter, Merck's hepatitis C drug, boceprevir, won expedited reviews by regulators in the U.S. and European Union. A decision by the U.S. Food and Drug Administration is also expected this year on an extended version of the Janumet diabetes pill, which combines Merck's Januvia with a generic drug called metformin.

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Vertex Loses $180M in Q4Vertex Pharmaceuticals Inc. reported a larger fourth-quarter loss as it spent more money to develop its hepatitis C drug candidate telaprevir, along with a potential cystic fibrosis treatment and other drugs. ...continue

2/2/11If Vertex Pharmaceuticals gets its way in talks with U.S. public health officials, most people over 50 could soon get blood tests to screen for hepatitis C infections at the doctor’s office. If the U.S. Centers for Disease Control and Prevention (CDC) agrees this is a good idea, it could prompt another 1 million patients who don’t realize they are infected to come out of the woodwork and start clamoring for Vertex’s new hepatitis C drug over the next few years./0.5/11Idenix Pharmaceuticals to Present at the 29th Annual J.P. Morgan Healthcare Conference

Vertex Pharmaceuticals Incorporated (Vertex) is engaged in the business of discovering, developing and commercializing small molecule drugs for the treatment of serious diseases. Telaprevir, the Company’s lead drug candidate, is an oral hepatitis C protease inhibitor and a new class of antiviral treatments in clinical development that target hepatitis C virus (HCV), infection. Telaprevir is being evaluated in a registration program focused on treatment-naïve and treatment-failure patients with genotype 1 HCV infection. VX-770, the lead drug candidate in its cystic fibrosis (CF), program is being evaluated in a registration program that focuses on patients with CF who have the G551D mutation in the gene responsible for CF. Vertex is conducting a number of Phase IIa clinical trials of its earlier-stage drug candidates. On March 12, 2009, Vertex acquired ViroChem Pharma Inc. (ViroChem)

Bayer teams up in IndiaGerman pharma and chemical group Bayer and Indian company Zydus Cadila have agreed to create a new company in India called Bayer Zydus Pharma. Each company will have a 50 per cent stake in the joint venture and equal representation on the management board. Bayer says it is looking to strengthen its position in the Indian pharma market. About 600 employees will transfer to the new company from the parent companies..

Teva buys Peruvian generics firmIsraeli generics firm Teva has said it will buy Corporacion Infarmasa, a pharmaceutical company based in Peru, from the two investment companies that own it. Teva already owns a company based in Peru: Corporacion Medco. Infarmasa makes generics, including corticosteroids, antihistamines, analgesics and antibiotics, at two sites in Lima. Financial details were not disclosed.

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Arena Pharmaceuticals: Should The CEO Go?By Ed Silverman // January 31st, 2011 // 10:37 amFor the past few months, Arena Pharmaceuticals ceo Jack Lief has been at the center of controversy over the extent to which the drugmaker’s Lorqess diet pill may have links to tumors in rats and, more specifically, whether data about this possibility was disclosed correctly. Last September, he was chastised for not conveying any info prior to disclosure that was made publicly by FDA staffers, who discussed the data in documents submitted for an FDA advisory panel

For example, consider just a few companies with new breakthrough drugs that are expected to become blockbusters (among nearly a dozen others): Dendreon’s (Nasdaq: DNDN ) prostate cancer drug, Provenge. Vertex Pharmaceuticals’ (Nasdaq: VRTX ) hepatitis therapy, Telaprevir. The drug is expected to receive FDA approval in May and could generate as much as $4 billion in annual sales. Human Genome Sciences’ (Nasdaq: HGSI ) treatment for lupus.But these “hotshot” analysts are also overlooking what I think will be the biggest growth area for biotech in the coming months…

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Week Of Jan 24

J&J Disappoints, 2011 Outlook WeakJohnson & Johnson's (JNJ - Analyst Report) fourth-quarter 2010 earnings (excluding special items) of $1.03 per share was in-line with the Zacks Consensus Estimate and a penny above the year-ago earnings of $1.02. Full year earnings came in at $4.76 per share, a penny above the Zacks Consensus Estimate and 2.8% above the year-ago earnings.

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Drugmaker Vertex moving to Fan PierCambridge-based Vertex Pharmaceuticals has signed a non-binding letter of intent for two buildings on Fan Pier, the company said.Under the terms of the deal, developer Joseph Fallon would build 1 million square feet of office and research and development space on the waterfront. Signing the lease would be contingent on Food and Drug Administration approval of Vertex’s drug to treat hepatitis C, according to Vertex spokesman Zachry Barber...read more

PRINCETON, N.J., Jan. 24, 2011 /PRNewswire/ -- Pharmasset, Inc. (Nasdaq: VRUS) announced today the underwriters of its recent underwritten public offering have exercised their option to purchase from Pharmasset an additional 495,000 shares of common stock.

May is going to be a solid month for drug approvals.The multiyear marathon between Schering-Plough and Vertex Pharmaceuticals (Nasdaq: VRTX) to develop the next-generation hepatitis C drug is finally coming to a close. Vertex announced yesterday that the Food and Drug Administration has accepted its New Drug Application to market telaprevir. Not surprisingly, given the unmet need, the agency will give telaprevir a priority review, so Vertex should hear back on or before May 23.Merck (NYSE: MRK), which took over development of boceprevir when it bought Schering-Plough, announced a few weeks ago that the FDA had accepted its application with a priority review, as well. Since one drug isn't all that material to Merck's business, the company didn't feel compelled to give an exact date of the submission, so we don't know exactly when the FDA will come back with a decision.

Bristol-Myers Squibb touted the five drugs in its pipeline that could reach the market by 2012; Pfizer highlighted its four most advanced oncology drugs; Merck & Co. emphasized its deep investment in cardiovascular drugs and the speedy filing of a New Drug Application for the hepatitis C drug boceprevir

have teamed up in a clinical partnership that marks the first cross-company collaboration to study two investigational oral drugs for the treatment of hepatitis C. The firms announced plans to study Bristol's NS5A replication complex inhibitor BMS-790052 and Pharmasset's nucleotide polymerase inhibitor on Jan. 10.Drug makers do not generally collaborate on clinical trials for investigative drugs for competitive reasons and tend instead to study combinations within their own pipelines or with at least one already marketed drug. Regulatory uncertainties and, perhaps, just as important, cultural resistance, also present hurdles. But novel-novel combinations increasingly are viewed as the best way to treat certain serious diseases, like cancer and hepatitis C, and have become more common.In hepatitis C specifically, the primary drug development focus is on combinations that could replace or reduce the use of the current standard of care, ribavirin and pegylated interferon (Roche's Pegasys or Merck's Pegintron), a regimen with a hasty side effect profile and limited efficacy. Bristol's decision to sign a clinical trial collaboration rather than in-licensing a polymerase inhibitor is intersting since the company recently acquired its hepatitsi C partner ZymoGenetics to gain more control over the program.

After the recalls of tens of millions of bottles and packages of numerous over-the-counter meds and surgical devices; months of negative publicity about corporate behavior; various government investigations and highly publicized congressional hearings, consumers may be turning away from Johnson & Johnson (JNJ) OTC products (back stories here, here, here and here).In the last quarter of 2010, Johnson & Johnson’s share of the $4.2 billion cough and cold market sank to about 5 percent from about 17 percent in early 2009, according to Nielsen data cited by Wells Fargo analyst Larry Biegelsen in an investor note. The J&J share of the $2.4 billion pain and arthritis market fell to just below 10 percent from just under 20 percent during the same period, while its share of the $1.4 billion gastrointestinal market slid to less than 5 percent from more than 10 percent.

Merck/Parexel International: Merck was one of the first companies to announce a strategy for bringing low-cost biologic copies to market after U.S. healthcare reform provided a clearer regulatory path for biosimilars. It announced the formation of a BioVentures unit in December 2008, with plans to put at least six biosimilars in development by 2012. It has kept close to the vest details of its target priorities, however, noting publicly only its interest in Amgen's granulocyte colony-stimulating factors Neupogen (filgrastim) and Neulasta (pegfilgrastim).Now, the Big Pharma has struck a partnership with clinical research organization Parexel International Corp. in which the CRO will conduct clinical trials for some of Merck's biosimilar programs. True to form, the companies won't say which therapeutic areas are covered by their collaboration, however. "One of the things that was exciting for us is that we were able to build a deal structure with Parexel in which they are rewarded for performance," Merck BioVentures President Michael Kamarck told "The Pink Sheet" DAILY. "In the biosimilars arena, time is everything, so...they will rpovide us with timeliness, and efficiency in execution of trials." -- Lisa LaMotta.

India is moving toward an innovative culture that will drive biotechnology’s continued growth. We are moving in the right direction – the Indian biotechnology sector’s prospects have never looked brighter. But there is more work that needs to be done. We need to increase collaborations and deal making across the biotech industry in India and globally, while promoting our assets and capabilities to global biotechnology and pharmaceutical companies as well as to investors and other financial stakeholders. Many biopharmaceutical companies want to diversify into generic drugs and biosimilars and are looking to India for partners based on our leadership in those segments.

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Biosimilars: Empty Threats?The Jan. 10 announcement by Novartis' Sandoz division of the start of Phase II trials of a biosimilar version of Roche's rituximab (Rituxan) is beginning to look a bit like posturing. After all, when was the last time a biosimilars firm gave away anything about its pipeline, let alone details of a specific compound?

By Deena BeasleySAN FRANCISCO Thu Jan 13, 2011 3:57pm ESTSAN FRANCISCO (Reuters) - More drugmakers are seeing potential in the business of producing copycat versions of expensive biotechnology drugs as U.S. guidelines take shape.The topic was at the forefront of plans discussed by several executives at this week's JP Morgan healthcare conference in San Francisco.Kevin Sharer, chief executive officer at Amgen Inc, said the world's largest maker of branded biotechnology drugs would consider entering the "biosimilars" space, particularly in emerging markets like Asia and South America.

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Should Merck Have Bought Schering-Plough?The merger has always been about much more than any one product. This merger was about taking two strong companies and making an even stronger one. As a result of the merger, the combined company is much better positioned to address the evolving needs of today’s healthcare environment:-Strong late-stage pipeline with boceprevir, odanacatib and anacetrapib, among others. [Matt adds: those are for hepatitis C, osteoporosis, and heart disease]

By Thomas Gryta Of DOW JONES NEWSWIRESNEW YORK (Dow Jones)--Vertex Pharmaceuticals Inc. (VRTX) Chief Executive Matthew Emmens is ready to go toe-to-toe with one of the world's biggest pharmaceutical companies, Merck & Co. (MRK).Cambridge, Mass., based Vertex has filed for Food and Drug Administration approval of telaprevir, which has shown a sharp increase in cure rates of hepatitis C in multiple studies when added to current treatments. Merck also has shown success with its similar drug, boceprevir, and has filed for approval with expedited review granted in both the U.S. and Europe.The drugs are expected to come to the market at similar times and create a market share battle, but Emmens isn't concerned about the size mismatch.

In its first move into the potentially lucrative Chinese market, Ligand Pharmaceuticals said Thursday that it partnered with the U.S. affiliate of a Hong Kong drug company to develop and market two experimental liver disease drugs in China..

(NASDAQ:VRTX) percentage change grew 0.36%, to close at $36.29 and its overall traded volume was 2.16M shares on Monday, the stock had average daily volume of 1.72M shares. VRTX opened at $35.95 and is trading within the range of $35.71-$36.54. The 52-week range of the stock is $31.25 – $43.94. The market capitalization of the company stands at $7.37B and it has 203.19M outstanding shares.Vertex Pharmaceuticals Incorporated (Vertex) is engaged in the business of discovering, developing and commercializing small molecule drugs for the treatment of serious diseases. Telaprevir, the Company’s lead drug candidate, is an oral hepatitis C protease inhibitor and a new class of antiviral treatments in clinical development that target hepatitis C virus (HCV), infection. Telaprevir is being evaluated in a registration program focused on treatment-naïve and treatment-failure patients with genotype 1 HCV infection. VX-770, the lead drug candidate in its cystic fibrosis (CF), program is being evaluated in a registration program that focuses on patients with CF who have the G551D mutation in the gene responsible for CF. Vertex is conducting a number of Phase IIa clinical trials of its earlier-stage drug candidates. On March 12, 2009, Vertex acquired ViroChem Pharma Inc. (ViroChem).,

On November 22, 2010, Vertex completed the submission of its New Drug Application (NDA) for telaprevir to the United States Food and Drug Administration (FDA). A response from the FDA regarding the company’s request for Priority Review of the telaprevir NDA is expected this month. The FDA’s goal for completion of its review for NDA submissions granted Priority Review status is six months from the NDA submission date

(BUSINESS WIRE) -- ---Cystic Fibrosis: First Phase 3 data for VX-770 expected in first quarter 2011- ---Additional ongoing trials in HCV, CF, epilepsy and rheumatoid arthritis- ---Vertex enters 2011 with cash and cash equivalents position of more than $1 billion- Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) today announced its 2011 business objectives in conjunction with the 29th Annual J.P. Morgan Healthcare Conference in San Francisco. Matthew Emmens, Chairman, President and Chief Executive Officer of Vertex, will discuss these objectives as part of a live webcast presentation, which will be available on Vertex's website, http://www.vrtx.com/ , on Monday, January 10 at 9:30 a.m. PT (12:30 p.m. ET)."2011 will be a landmark year for Vertex as we prepare for the expected launch of telaprevir in hepatitis C and advance other new therapies in development," said Mr. Emmens.

Chiva Pharmaceuticals in-licensed China rights for two clinical-stage liver drugs from Ligand Pharma (NSDQ: LGND) (see story). It also will receive non-exclusive rights to Ligand’s HepDirect technology to discover and develop new molecules for hepatitis B, hepatitis C and hepatocellular carcinoma. Chiva, which is headquartered in California, is an affiliate of Hainan Kaihua Pharma.

January 2011 00:20Inhibitex, Inc. today reported positive preliminary interim safety and antiviral data from the first two monotherapy cohorts of its ongoing Phase 1b clinical trial of INX-189, an oral NS5b nucleotide inhibitor being developed to treat chronic infections caused by hepatitis C virus (HCV).

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Week Of Jan 3rd

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The U.S. Food and Drug Administration accepted Merck's approval filing for its hepatitis C drug boceprevir, the company said Thursday. The news from Merck is a bit of a surprise since the company hadn't previously announced or confirmed that boceprevir had been filed with U.S. regulators. It's also a tad embarrassing for Vertex, which is still waiting to hear back from FDA about the acceptance of the approval filing for telaprevir, its competing hepatitis C drug. That news should come by Jan. 24, based on Vertex's previous announcement that it filed the drug's application on Nov. 23. Still, it seems as if Merck beat Vertex to the FDA by at least two weeks.Continue Reading....

Pharmasset Reports Positive Results from its HCV Clinical Programs - PSI-7977 400mg QD with pegylated interferon and ribavirin was generally safe and well tolerated while demonstrating potent viral suppression in patients with HCV genotype 2 or 3 over 12 weeks of dosing- PSI-938 300mg QD monotherapy was generally safe and well tolerated over 14 days of dosing and demonstrated potent antiviral activity and no viral breakthrough

BASi (Bioanalytical Systems Inc.), a life sciences company in the Purdue Research Park, has entered into a Preferred Provider Agreement (PPA) with Princeton, New Jersey-based Pharmasset Inc., a clinical-stage pharmaceutical company committed to discovering, developing and commercializing novel drugs to treat viral infections, to provide preclinical services for pre-IND and post-IND activities. The agreement includes provisions to provide exclusive toxicology services as well as pharmaceutical analysis and bioanalytical services as needed.

Chiva Pharmaceuticals negotiated Chinese development rights to Ligand Pharmaceuticals’ clinical-stage HepDirect candidates, Pradefovir for hepatitis B (hepB) and MB01733 for hepatocellular carcinoma (HCC). The deal also gives Chiva a nonexclusive license to use Ligand’s HepDirect technology for the discovery, development, and worldwide commercialization of new candidates against hep B, hep C, and HCC.

Pradefovir is a HepDirect prodrug of PMEA, the active metabolite in the FDA-approved HepB drug adefovir dipivoxil (Hepsera®). The drug is currently in Phase II development in the U.S. MB07133 is a HepDirect prodrug of the intermediate form of cytarabine (araC) 5’-monophosphate. Currently in Phase I/II development treatment of hepatocellular carcinoma, the drug has already demonstrated strong response rates in terms of intrahepatic tumor regression in a U.S. trial, Ligand states.

The supply of the Hepatitis C drug to Vertex by Shasun Chemicals is set to take off earlier than anticipated. The product is set to launch in June-July next year. "Commercial supplies have already started and we are preparing for the launch this year," says Vimal Kumar, Managing Director of Shasun Chemicals, in an exclusive interview with CNBC-TV18's Udayan Mukherjee and Mitali Mukherjee.

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Pharmasset to Present at the 29th Annual J.P. Morgan Healthcare Conference,PRINCETON, N.J., Jan. 4, 2011 /PRNewswire/ -- Pharmasset, Inc. (Nasdaq: VRUS) announces that management will present at the 29th Annual J.P. Morgan Healthcare Conference to be held January 10-13, 2011 at the Westin St. Francis Hotel, San Francisco, CA. Schaefer Price, President and Chief Executive Officer, will provide an overview of the company on Wednesday, January 12, 2011 at 9:00 AM (PT).To access a simultaneous webcast of Mr. Price's overview via the internet, log on to the "Events & Presentations" section of the Investor Center on Pharmasset's website at http://investor.pharmasset.com/events.cfm. Please connect to the website at least ten minutes prior to the start of the presentation to ensure adequate time for a reliable connection and any software download that may be necessary for the webcast.A replay of the webcast will be available on Pharmasset's website for thirty days following the conference. The investor presentation will be available for download in PDF format immediately following the presentation in the "Events & Presentations" section of the Investor Center on Pharmasset's website at http://investor.pharmasset.com/events.cfm.

After the bell Tuesday, Anadys Pharmaceuticals, Inc. (Nasdaq:ANDS) announced that it has initiated the planned Phase IIb study of ANA598 in combination with pegylated interferon and ribavirin. The protocol for the study has been cleared by the United States Food and Drug Administration and Health Canada. Patient screening has begun and patient dosing is expected to commence within the next several weeks. In the study ANA598 will be tested in both treatment-naive patients and treatment-experienced patients who failed a prior course of therapy with interferon and ribavirin. ANA598 is the Company's direct-acting antiviral, or DAA, being developed for the treatment of hepatitis C.Earlier Tuesday: Shares of XOMA Ltd. (Nasdaq:XOMA) have caught fire once again. Today the company announced a partnership with Les Laboratoires Servier (Servier), France's largest privately-held pharmaceutical company, to develop and commercialize XOMA 052, XOMA's anti-inflammatory drug candidate, in multiple indications. The drug is designed to inhibit the pro-inflammatory cytokine interleukin-1 beta that is believed to be a primary trigger of pathologic inflammation in multiple diseases.

InterMune, Inc. (NASDAQ:ITMN) decreased -0.34%, currently trading at $37.78 and its overall traded volume was 1.04M shares while reporting, against its average volume of 2.44M. ITMN opened the day at $37.64, it made an intraday low of $36.98 and an intraday high of $38.58. The stock has a 52 week low of $8.34 and 52 week high of $49.46. ITMN's market capitalization is 2.12B and it has 56.03M outstanding shares. InterMune, Inc. (InterMune) is a biotech company focused on developing and commercializing therapies in pulmonology and hepatology. In November 2008, InterMune together with Roche and Pharmasset, Inc. (Pharmasset) announced the initiation of INFORM-1, a dual combination clinical trial investigating the combination of two oral antiviral molecules in the absence of interferon. In August 2009, the Company together with Hoffmann-LaRoche Inc. and F. Hoffmann-LaRoche Ltd. (collectively Roche) began a Phase IIb study testing RG7227 in combination with PEGASYS (peginterferon alfa-2a) and COPEGUS (ribavirin), the standard of care (SOC) in hepatitis C virus (HCV).

Vertex Pharmaceuticals Incorporated (Vertex) is engaged in the business of discovering, developing and commercializing small molecule drugs for the treatment of serious diseases. Telaprevir, the Company’s lead drug candidate, is an oral hepatitis C protease inhibitor and a new class of antiviral treatments in clinical development that target hepatitis C virus (HCV), infection. Telaprevir is being evaluated in a registration program focused on treatment-naïve and treatment-failure patients with genotype 1 HCV infection. VX-770, the lead drug candidate in its cystic fibrosis (CF), program is being evaluated in a registration program that focuses on patients with CF who have the G551D mutation in the gene responsible for CF. Vertex is conducting a number of Phase IIa clinical trials of its earlier-stage drug candidates. On March 12, 2009, Vertex acquired ViroChem Pharma Inc. (ViroChem).Shares of Vertex Pharmaceuticals (NASDAQ: VRTX) traded down 0.79% during mid-day trading on Friday, hitting $35.06. Vertex Pharmaceuticals has a 52 week low of $31.25 and a 52 week high of $44.24. The stock’s 50-day moving average is $34.28 and its 200-day moving average is $34.94. On average, analysts predict that Vertex Pharmaceuticals will post $-0.90 EPS next quarter. The company has a market cap of $7.124 billion and a price-to-earnings ratio of N/A.For more information about Zacks Investment Research’s equity research offerings, visit Zacks.com.

Zacks Investment Research research analysts reiterated a “neutral” rating for shares of Achillion Pharmaceuticals Inc (NASDAQ: ACHN) in a research note issued to investors on Thursday.

Achillion Pharmaceuticals Inc (NASDAQ: ACHN)’s stock traded down 1.20% on Monday, hitting $4.10. Achillion Pharmaceuticals Inc has a 52 week range of $1.99 to $4.20. The stock’s 50-day moving average is $3.1 and its 200-day moving average is $2.77. Analysts predict on average that Achillion Pharmaceuticals Inc will post $-0.13 earnings per share next quarter. The company has a market cap of $239.2 million and a P/E (price-to-earnings ratio) of N/A.About Achillion Pharmaceuticals Inc (NASDAQ: ACHN)Achillion Pharmaceuticals, Inc. (Achillion) is a biopharmaceutical company focused on the discovery, development and commercialization of treatments for infectious diseases. Within the anti-infective market, the Company focuses on the development of antivirals for the treatment of chronic hepatitis C and the development of antibacterials for the treatment of resistant bacterial infections. As of December 31, 2009, Achillion focused on developing three Hepatitis C virus (HCV) drug candidates: ACH-1625, a protease inhibitor for the treatment of chronic hepatitis C in phase Ib clinical testing; ACH-1095, a NS4A antagonist also for the treatment of chronic hepatitis C, in late stage preclinical testing, and ACH-2684, a high-potency protease inhibitor in preclinical testing. In addition, it has established other product candidates, which include ACH-702 for the treatment of serious bacterial infections and elvucitabine for the treatment of human immunodeficiency virus (HIV) infection.For more information about Zacks Investment Research‘s equity research offerings, visit Zacks.com.

“There has been no systemic change in how the FDA is approaching drug approvals” Walsh said in an e-mail. “The primary factor driving first-cycle approval is the quality of the application.”No drugmaker had more than one new medicine approved in 2010, according to data Bloomberg compiled. The figures, derived from an agency database of “new molecular entities,” exclude blood products and vaccines such as Seattle-based Dendreon Corp.’s Provenge for prostate cancer, approved April 29 after a three-year wait, because the FDA doesn’t consider them drugs.

Merck, of Whitehouse Station, N.J., made progress in 2010 launching new products and advancing its drug pipeline, said Ron Rogers, a spokesman. Products to be evaluated this year include boceprevir, a hepatitis C treatment that five analysts project, on average, will have sales of $524.4 million in 2014.

Dynavax Technologies Corporation (Dynavax) is a biopharmaceutical company that discovers and develops products to prevent and treat infectious diseases, asthma and inflammatory and autoimmune diseases. The Companyâ€™s principal product candidate is HEPLISAV, a Phase III investigational adult hepatitis B vaccine. Its pipeline of product candidate includes HEPLISAV; its Universal Flu vaccine; clinical-stage programs for hepatitis C and hepatitis B therapies, and preclinical programs partnered with AstraZeneca and GlaxoSmithKline (GSK). In January 2010, the Company announced the completion of the acquisition of Symphony Dynamo, Inc.Dynavax Technologies Co. (NASDAQ: DVAX) traded down 3.44% during mid-day trading on Monday. The stock has a 52 week low of $1.19 and a 52 week high of $3.24. Its 50-day moving average is $2.38 and its 200-day moving average is $2.01. The company has a market cap of $357.1 million and a price-to-earnings ratio of N/A.Stay on top of analysts' coverage with American Banking & Market News'daily email newsletterthat provides a concise list of analysts’ upgrades, analysts’ downgrades and analysts’ price target changes for each day.Click Here to register..

This is supposed to be Cambridge-based Vertex Pharmaceuticals’ big year. The company has been steadily adding staff over the past year and a half, as the company gears up for its anticipated transformation to a profitable, commercial biopharmaceutical company, joining the elite ranks of its neighbors Biogen Idec and Genzyme Corp.Vertex submitted its much-anticipated drug candidate Telaprevir, for the treatment of hepatitis C to the U.S. Food and Drug Administration in November. Vertex has asked for a priority review, meaning that a decision could come as soon as May.“This submission is a milestone in our more than 15-year effort to change the way hepatitis C is treated,” said Matthew Emmens, chairman, president and CEO of Vertex, at the time. But the company has recently halted two other, earlier stage clinical trials testing Telaprevir in conjunction with its other potential hepatitis C therapy called VX-222, because the combination wasn’t working. Three other trials, using Telaprevir in combination with other, non-Vertex drugs, are ongoing.The stakes are high. Howard Liang, an analyst at Boston-based investment bank Leerink Swann has estimated that telaprevir may generate $2.6 billion annually by 2013, if approved. While its partners, Belgium-based Tibotec Pharmaceuticals and Japan-based Mitsubishi Tanabe Pharma hold the rights to commercialize the drug outside the United States, it is the American market that is expected to be the biggest revenue driver for Telapre ...This is supposed to be Cambridge-based Vertex Pharmaceuticals’ big year. The company has been steadily adding staff over the past year and a half, as the company gears up for its anticipated transformation to a profitable, commercial biopharmaceutical company, joining the elite ranks of its neighbors Biogen Idec and Genzyme Corp.Vertex submitted its much-anticipated drug candidate Telaprevir, for the treatment of hepatitis C to the U.S. Food and Drug Administration in November. Vertex has asked for a priority review, meaning that a decision could come as soon as May.“This submission is a milestone in our more than 15-year effort to change the way hepatitis C is treated,” said Matthew Emmens, chairman, president and CEO of Vertex, at the time. But the company has recently halted two other, earlier stage clinical trials testing Telaprevir in conjunction with its other potential hepatitis C therapy called VX-222, because the combination wasn’t working. Three other trials, using Telaprevir in combination with other, non-Vertex drugs, are ongoing.The stakes are high. Howard Liang, an analyst at Boston-based investment bank Leerink Swann has estimated that telaprevir may generate $2.6 billion annually by 2013, if approved. While its partners, Belgium-based Tibotec Pharmaceuticals and Japan-based Mitsubishi Tanabe Pharma hold the rights to commercialize the drug outside the United States, it is the American market that is expected to be the biggest revenue driver for Telaprevir.A blockbuster drug approval in 2011 would also raise the profile of Vertex as an attractive acquisition target, analysts say, as big pharma companies seek to boost their supplies of new approved drugs to replace those that are going off patent.Julie M. Donnelly can be reached at juliedonnelly@bizjournals.com.Read Full Article

At the other extreme, Vertex Pharmaceuticals' (Nasdaq: VRTX) hepatitis C treatment, telaprevir, seems like a shoo-in. Given the extensive data and unmet need -- current treatments only cure about half of patients -- an approval seems all but certain.Keeping in mind that you still have to expect the unexpected, of course.Keep up with the drugs facing FDA decisions using The Fool's My Watchlist feature or subscribe to Brian Orelli's RSS feed here.

. ,Vertex Pharmaceuticals (NASDAQ: VRTX) Given “Neutral” Rating by Zacks Investment Research AnalystsDecember 31st, 2010 • Equities research analysts at Zacks Investment Research reiterated a “neutral” rating on shares of Vertex Pharmaceuticals (NASDAQ: VRTX) in a research note to clients and investors on Monday.Separately, analysts at Canaccord Genuity raised their price target on shares of Vertex Pharmaceuticals to $35.00 in a research note to investors on Tuesday, December 21st. They now have a “hold” rating on the stock.Vertex Pharmaceuticals Incorporated (Vertex) is engaged in the business of discovering, developing and commercializing small molecule drugs for the treatment of serious diseases. Telaprevir, the Company’s lead drug candidate, is an oral hepatitis C protease inhibitor and a new class of antiviral treatments in clinical development that target hepatitis C virus (HCV), infection. Telaprevir is being evaluated in a registration program focused on treatment-naïve and treatment-failure patients with genotype 1 HCV infection. VX-770, the lead drug candidate in its cystic fibrosis (CF), program is being evaluated in a registration program that focuses on patients with CF who have the G551D mutation in the gene responsible for CF. Vertex is conducting a number of Phase IIa clinical trials of its earlier-stage drug candidates. On March 12, 2009, Vertex acquired ViroChem Pharma Inc. (ViroChem).Shares of Vertex Pharmaceuticals (NASDAQ: VRTX) traded down 0.79% during mid-day trading on Friday, hitting $35.06. Vertex Pharmaceuticals has a 52 week low of $31.25 and a 52 week high of $44.24. The stock’s 50-day moving average is $34.28 and its 200-day moving average is $34.94. On average, analysts predict that Vertex Pharmaceuticals will post $-0.90

(Public,NASDAQ:ANDS) increased 2.07%, to close at $1.48 and its overall traded volume was 702,093.00 shares during the last session against its average volume of 882,279.00. ANDS opened the day at $1.55, it made an intraday low of $1.46 and an intraday high of $1.55. The stock has a 52 week low of $0.90 and 52 week high of $3.24. ANDS's market capitalization is 84.51M and it has 57.10M outstanding shares.About Anadys Pharmaceuticals, Inc. (Public,NASDAQ:ANDS)Anadys Pharmaceuticals, Inc. is a biopharmaceutical company engaged in developing medicines for the treatment of hepatitis C. The Company is developing ANA598, a small-molecule, non-nucleoside inhibitor of the NS5B polymerase for the treatment of hepatitis C. It has also investigated ANA773, an oral, small-molecule inducer of endogenous interferons that acts via the Toll-like receptor 7 (TLR7), pathway in a Phase I trial in hepatitis C. In June 2009, the Company initiated a strategic restructuring to focus its operations on the development of ANA598, in particular the Phase II study of ANA598 in combination with pegylated interferon and ribavirin. As part of the restructuring, it suspended further development of ANA773 Continue reading.....

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Vertex Pharmaceuticals Incorporated

(Public,NASDAQ:VRTX) decreased -0.37%, to close at $35.34 and its overall traded volume was 630,062.00 shares during the last session against its average volume of 1.78M. VRTX opened the day at $35.39, it made an intraday low of $35.22 and an intraday high of $35.49. The stock has a 52 week low of $31.25 and 52 week high of $44.24. VRTX's market capitalization is 7.18B and it has 203.19M outstanding shares.About Vertex Pharmaceuticals Incorporated (Public,NASDAQ:VRTX)Vertex Pharmaceuticals Incorporated (Vertex) is engaged in the business of discovering, developing and commercializing small molecule drugs for the treatment of serious diseases. Telaprevir, the Company’s lead drug candidate, is an oral hepatitis C protease inhibitor and a new class of antiviral treatments in clinical development that target hepatitis C virus (HCV), infection. Telaprevir is being evaluated in a registration program focused on treatment-naïve and treatment-failure patients with genotype 1 HCV infection. VX-770, the lead drug candidate in its cystic fibrosis (CF), program is being evaluated in a registration program that focuses on patients with CF who have the G551D mutation in the gene responsible for CF. Vertex is conducting a number of Phase IIa clinical trials of its earlier-stage drug candidates. On March 12, 2009, Vertex acquired ViroChem Pharma Inc. (ViroChem).

ACH-1625 has already been shown to significantly reduce viral loads in patients by 3 to 4.25 log10 and maintain a sustained viral response even after therapy has stopped. It has also been shown to be safe up to 2000mg/day, a level far above the highest dose tested in this Phase IIa.Pre-clinical studies show ACH-1625 to be additive-synergistic to ribavirin and peg-interferon alpha; this study will be a first look at how well those studies translate in the clinic. Although 28 days is still short, the multiple active drug doses as well as a placebo control will allow investigators to gauge in detail the effectiveness of ACH-1625 in this combination. In any case, a full profile of the compound and its potential as part of this three-drug regimen will be in known by the end of 2011. There’s a lot riding on this study.Aside from its lead compound, Achillion also has a lineup of several other HCV antivirals in development. The once promising ACH-1095, an inhibitor of the NS4A protease is in Phase I. It was originally developed in collaboration with Gilead (GILD), but its rights have since been handed back to the company- though Gilead still retains the ability to opt in at a later stage in the compound’s development.

Debrianna Obara, VP, media, Razorfish HealthIn the digital world of health, more and more brands will focus on distribution of content as opposed to straight display media or search buys. This shift will allow brands who have invested in educational and branded content to amortize that investment by

getting more health seekers to interact with that content. The user benefits, as they can interact with valuable information without leaving their trusted 3rd party website of choice (such as AOL/Yahoo!/WebMD).

By contrast, growth of just 1% to 3% is forecast for the major European markets and Canada, with 3% to 5% growth predicted in the United States, which is still the world's largest drug consumer with 2011 sales expected to hit $320-$330 billion, up from $310 billion this year.A tougher regulatory environment in the developed countries. The U.S. Food & Drug Administration and many foreign regulatory bodies are becoming more reluctant to grant initial approval for new drugs in the face of numerous recalls and growing safety concerns among the public.Pressure from governments and private insurers to reduce medical and drug costs. Governments in virtually all of the developed countries are pushing cost-cutting measures, including increased use of generic medications, mandatory price cuts for brand-name drugs and elimination of rebates. Private insurers are increasing requirements for pre-authorization of treatments and higher cost-sharing percentages for patients.

Vertex's Telaprevir Aims to Revolutionize HCV TreatmentVertex (VRTX) is close to commercializing its hepatitis C drug telaprevir, part of a new class of therapies that have the potential to revolutionize the way the disease is treated. HCV's current standard of care requires 48 weeks of treatment, causes terrible side effects, and is still only able to cure patients 50% of the time. In contrast, telaprevir has the potential to cut treatment time in half and double cure rates. The drug is widely regarded as a major advancement, and we think telaprevir could hit the market in the first half of next year.

Vertex's chief rival, Merck (MRK), is developing a protease inhibitor of its own. We think telaprevir's slightly superior efficacy and convenience profile should give it an advantage with prescribers. However, Vertex has no commercialization experience, and Merck's established salesforce and ability to package the drug with the current standard of care should help even the commercialization playing field. Vertex ranked number 1 on our 2010 biotech takeout list, and we continue to believe the firm would benefit from the sales know-how of a larger pharma player like current partner Johnson & Johnson (JNJ). The stock currently trades at a 25% discount to our fair value estimate, providing an attractive entry point for investors with a stomach for risk.

Predictions for 2011Biotech and the Capital Markets: The biotech industry did benefit from the return of investor confidence in the second half of the year...with the Burrill Biotech Select Index outperforming the Dow Jones Industrial Average on an annual basis. Expect to see the biotech industry continue to outperform the general markets as the financing environment continues to improve in 2011.

Biotech IPOs: The biotech IPO window will remain open despite the fact that the 17 new biotech issues that debuted on the U.S. market in 2010 were plagued by lackluster receptions (selling fewer shares well below the pricing range) and their average annual market performance was down by 13 percent. By the end of 2011, I predict that at least 25 biotech IPOs, possibly more, in the US will be completed.Capital: The industry has achieved a "steady state" in terms of financing...raising about $15 billion annually. This situation will continue in 2011. The industry's collective market cap will also remain at its present $360 billion level as market value growth will be offset by acquisitions.Partnering: There will be no major slow down in big Pharma's appetite for biotech partnering. Both big pharma and big biotech will again compete for companies with advanced product pipelines. The deal structures will embrace "shared risk". The days when biotech enjoyed major upfront payments for pharma companies to access their technologies are over. Collaborations with emerging market players in China, India and Latin America will also increase.Mergers & Acquisitions: The predicted surge in big Pharma acquisitions of biotech companies did not happen in 2010. It will in 2011. Sanofi will finally seal the deal and acquire Genzyme and this will usher in several other marquee acquisitions of blue chip biotech companies.Pharma Restructuring: Pharma companies will continue to make job cuts and restructuring their businesses ahead of loss of patents protection on major blockbusters.Increased government involvement on healthcare: The federal government, through Medicare and Medicaid, will continue to play a greater role in the delivery and reimbursement of healthcare. This trend will create an array of new regulatory and compensatory rules, issues, and challenges for healthcare providers.Biosimilars: Healthcare reform carries provision instructing the FDA to create a pathway for biosimilars and expect to see both biotech and pharma companies take a keen interest in the discussions on the drafting of new regulations governing the development of biosimilars.Converging technologies impact healthcare: Expect to see a greater emphasis on prevention and wellness. A greater understanding of human genomics and the advent of molecular diagnostics, and the convergence of information, wireless, and medical technology promises to make personalized medicine an ever present reality in the way doctors and patients approach healthcare.Regulatory environment: The industry will continue to adjust to a regulatory environment that includes comparative effectiveness research (CER). Payors will be looking at CER as a way to gather the necessary data on whether to reimburse for genomically guided medicines. With PDUFA expiring in 2012 there will be a major battleground over drug safety and review issues in Congress in 2011.Science and Technology: The evolving legal battle over the patentability of genes will heat up; uncertainty will continue to swirl around stem cells but regenerative medicine will be hot.Clean tech will boom: The clean tech boom in non-food crops will continue as major investments in solar power, wind power, and next generation biofuels gets attention. The market will not only embrace green, but technologies that improve energy efficiency and environmental "friendliness"—less polluting and less consuming.Global markets: Expect emerging markets to continue to be a dominant factor. Increasing affluence, a growing middle class, and government policies will make healthcare big business in these countries.Continue Reading....

Wednesday, December 22, 2010Vertex halts part of HCV trialVertex Pharmaceuticals Inc. is discontinuing part of a Phase 2 clinical trial of its hepatitis C virus (HCV) drug candidate. The Cambridge biotech halted the second two-drug treatment, which consisted of telaprevir and VX-222, though it continues with three-drug and four-drug regimen trials.

HCV Education
Review learning activities, editorials, with new data about interferon-free regimens approved for HCV, as well as investigational drugs still in the pipeline. Links are provided to support, patient friendly information, clinical trials, peer-reviewed journals, videos, conferences with commentary, all updated on a continuous basis.

Users can search for a hepatitis C clinical trial by category (genotype), or learn how to evaluate a clinical trial and become familiar with commonly used terms. HCV Advocate offers an easy to navigate HCV Medications Blog as well, organized by HCV genotype

View each rebuttal and all ongoing media coverage. In June the HCV community was blindsided when an article with a somewhat "clickbait" headline was released by The Guardian. The Guardian reported on a systematic review published by the Cochrane Collaboration that suggested achieving SVR (cure) for patients using hepatitis C direct-acting antivirals (DAAs) doesn't correlate with any long term benefits.

The controversy over expensive new drugs for hepatitis C
Link to research and news articles addressing the high cost of hepatitis C drugs; insurance restrictions - private insurers/Medicaid - and availability of generic versions/India, Egypt and other lower-income countries or through online "buyers clubs"

Liver Cancer After Treatment For Hepatitis C
​Research demonstrates that while SVR markedly reduced liver-related complications and liver cancer, some long-term risk for liver cancer remained in those who were cured of Hepatitis C. But after direct-acting antiviral therapy does the risk of developing liver cancer increase?

Meeting Updates

April 19-23
The European Association for the Study of the Liver (EASL) International Liver Congress (ILC 2017)

Merck today announced its strategic decision to discontinue the development of the investigational combination regimens MK-3682B (grazoprevir/ruzasvir/ uprifosbuvir) and MK-3682C (ruzasvir/uprifosbuvir) for the treatment of chronic hepatitis C virus (HCV) infection. This decision was made based on a review of available Phase 2 efficacy data and in consideration of the evolving marketplace and the growing number of treatment options available for patients with chronic HCV infection

Two HCV Drugs to Be Discontinued
The Food and Drug Administration (FDA) announced that Rebetol(ribavirin; Merck) capsules and PegIntron (peginterferon alfa-2b; Merck) for Injection are being discontinued. The decision is business-related and not due to safety or efficacy issues with the drugs.

Rebetol is a nucleoside analogue indicated for chronic hepatitis C in combination with interferon alfa-2b (pegylated and nonpegylated), in patients ≥3 years of age with compensated liver disease. It is supplied as 200mg capsules in 56-, 70-, and 84-count bottles. The Rebetol discontinuation is effective February 1, 2016.
PegIntron is an antiviral indicated for treatment of chronic hepatitis C in patients with compensated liver disease. It is supplied as 50mcg/0.5mL, 80mcg/0.5mL, 120mcg/0.5mL, and 150mcg/0.5mL single-use vials and single-use pre-filled pens. No effective date is available for the PegIntron discontinuation.

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